The Food and Drug Administration (FDA) today proposed significant regulatory changes to make experimental drugs more widely and easily available to seriously ill patients with no other treatment options and to clarify the circumstances and the costs for which a manufacturer can charge for an experimental drug.

Under the proposed rule, expanded access for experimental drugs would be available to individual patients, small patient groups, and larger populations under a treatment plan when there is no satisfactory alternative therapy to diagnose, monitor or treat the disease or condition.

"This proposed reform is carefully designed to balance several objectives," said Dr. Andrew C. von Eschenbach, Acting FDA Commissioner. "One goal is to enable many more patients who lack satisfactory alternatives to have access to unapproved medicines, while balancing the need for safeguarding the individual patient. Another equally important goal is to ensure the continued integrity of the scientific process that brings safe and effective drugs to the market."

"FDA hopes this proposal will increase awareness in the healthcare community of the range of options available for obtaining experimental drugs for seriously ill patients," added Dr. Janet Woodcock, FDA's Deputy Commissioner for Operations. "By clarifying and streamlining the processes, FDA also hopes to encourage companies to make such drugs available, and reduce barriers for healthcare practitioners in obtaining them."

FDA has allowed many types of access to experimental therapies since the 1970's. Some of the larger programs, including those under the treatment IND (Investigational New Drug) regulations, were successful in enabling tens of thousands of patients with HIV/AIDS, cancer and cardiovascular diseases to receive promising therapies before the products were approved for marketing. However, the existing regulations did not adequately describe the full range of programs available, explicitly recognizing only emergency use for individual patients and widespread treatment use access for large groups of patients. FDA believes it is important that its regulations clearly reflect the full range of treatment use programs available to ensure broad and equitable access to experimental drugs for treatment use. The regulations covering when it is appropriate to charge for an experimental drug need revisions because they fail to account for the full range of circumstances in which charging should be permissible and because they have proven difficult to interpret in practice, resulting in confusion over what costs could be recovered.

The proposed rules, which are open for comment for 90 days are described in detail at the following CDER web address: (http://www.fda.gov/cder/regulatory/applications/IND_PR.htm) The most significant proposals would:

(1) Modernize applicable regulations to include all circumstances under which access to experimental drugs is permitted, including:

single patients in non-emergency and emergency settings;

small groups of patients; and

larger groups of patients under a treatment IND. To authorize these expanded access treatment uses, FDA generally must be satisfied that the patient's serious or immediately life-threatening disease or condition has no satisfactory approved therapy; that the potential benefit for the patient justifies the potential risks; and that providing the therapy will not interfere with the drug's development.

(2) Make experimental drugs more widely available in appropriate situations by establishing criteria that link the level of evidence needed to support the use of an experimental drug to the seriousness of the disease and the number of patients likely to be treated with the drug;

(3) Revise the current regulation regarding manufacturers' recovery of the costs of an experimental drug to:

clarify that such charges are permissible in a clinical trial only to facilitate development of drugs that promise significant advantages over existing therapies, and might not otherwise be developed because of their high cost;

clarify that allowing charging for treatment use of an experimental drug is intended to facilitate and encourage access to drugs that might not be made available for treatment use unless a manufacturer is able to recover its costs.

The proposal also would simplify the cost recovery calculation by making clear that charges for an experimental drug used in a clinical trial may include only direct costs associated with the drug's development, and that charges for experimental drugs for treatment use may also include administrative costs of making the drug available for intermediatepatient populations and under large scale treatment INDs.

Written comments, identified by Docket No. 2006N-0062 and RIN 0910-AF14 (for expanded access proposals) and Docket No. 2006N-0061 and/or RIN 0910-AF13, (for cost recovery proposals), may be submitted by any of the following methods: